UMass Chan licenses gene therapy for rare genetic disorder to Raiden Science Foundation
Raiden Pham, who was diagnosed with UBA5 disorder in 2021, with his mother Linda Pham. Photo courtesy of Tommy and Linda...
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New hope for children with devastating rare genetic disorder, thanks to world-first research in Manchester
The parents of a three-year-old boy born with a devastating, life-limiting genetic condition say they are now excited for his...
Researchers to test gene therapy for rare diseases with a new FDA trial protocol
Schematic of “umbrella of umbrellas” master protocol clinical trial. Credit: The American Journal of Human Genetics (2025). DOI: 10.1016/j.ajhg.2025.10.006 Earlier...
Gene therapy trial offers breakthrough for rare genetic disorder
Credit: Pixabay/CC0 Public Domain A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising...
World-first CRISPR therapy could ‘transform’ treatment for rare genetic diseases, but key challenges lie ahead
It has been described as a revolutionary technology — and won its inventors a Nobel Prize.CRISPR gene editing, often simply...
Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment
News ReleaseThursday, May 15, 2025 NIH-supported gene-editing platform lays groundwork to rapidly develop treatments for other rare genetic diseases.A research...
Dallas scientists granted $420K to test gene therapy for rare neurological disease
Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by age...
The Future of Gene-Editing Treatments for Rare Diseases < Yale School of Medicine
Angelman syndrome is a rare genetic disorder caused by a mutation on chromosome 15, which hinders the production of a...
Gene therapy shows promise for treating rare muscle disease
A University of Alberta research team has taken the first step toward proving its innovative gene therapy can successfully treat...
